Intraventricular Delivery of siRNA Nanoparticles to the Central Nervous System
Open accessAbstract
Alzheimer's disease (AD) is a progressive neurodegenerative disease currently lacking effective treatment. Efficient delivery of siRNA via nanoparticles may emerge as a viable therapeutic approach to treat AD and other central nervous system disorders. We report here the use of a linear polyethyleneimine (LPEI)-g-polyethylene glycol (PEG) copolymer-based micellar nanoparticle system to deliver siRNA targeting BACE1 and APP, two therapeutic targets of AD. Using LPEI-siRNA nanoparticles against either BACE1 or APP in cultured mouse neuroblastoma (N2a) cells, we observe selective knockdown, respectively, of BACE1 or APP. The encapsulation of siRNA by LPEI-g-PEG carriers, with different grafting degrees of PEG, leads to the formation of micellar nanoparticles with distinct morphologies, including worm-like, rod-like, or spherical nanoparticles. By infusing these shaped nanoparticles into mouse lateral ventricles, we show that rod-shaped nanoparticles achieved the most efficient knockdown of BACE1 in the brain. Furthermore, such knockdown is evident in spinal cords of these treated mice. Taken together, our findings indicate that the shape of siRNA-encapsulated nanoparticles is an important determinant for their delivery and gene knockdown efficiency in the central nervous system.
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Rod-shaped micellar nanoparticles encapsulating siRNA targeting BACE1 and APP showed the highest efficiency in reducing BACE1 levels following intracerebroventricular administration.
“Notably, rod-shaped nanoparticles showed the highest efficiency in reducing BACE1 levels following intracerebroventricular administration [114].”
From Targeted Nanoparticles for Drug Delivery Across the Blood–Brain Barrier in Early and Late Stages of Alzheimer's Disease: A Review (2026) · cites this paper as [114]
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- Authors
- Shyam, R., Ren, Y., Lee, J., Braunstein, K. E., Mao, H.-Q., & Wong, P. C.
- Affiliations
- Johns Hopkins University (US)
- Corresponding author
- Mao, Hai-Quan
- Wong, Philip C
- Venue
- Molecular Therapy - Nucleic Acids
- Publisher
- Elsevier BV
- Published
- 2015
- Volume
- 4
- Pages
- e242
- ISSN
- 2162-2531 (ISSN-L)
- OpenAlex
- W2173365046
- Cited by
- 58
- References
- 57
- Citations by year
- 2026: 3 · 2025: 4 · 2024: 8 · 2023: 11 · 2022: 6 · 2021: 5 · 2020: 3 · 2019: 6 · 2018: 6 · 2017: 1 · 2016: 5
- FWCI
- 1.74
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Nucleic Acids (publishedVersion, cc-by-nc-sa)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- National Institute of General Medical Sciences (T32GM007057)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Physical Sciences › Materials Science › Polymers and Plastics › Dendrimers and Hyperbranched Polymers
- Keywords
- siRNA delivery, Alzheimer's disease, BACE1, amyloid precursor protein, micellar nanoparticles, nanoparticle morphology, rod-shaped nanoparticles, gene knockdown, central nervous system, lateral ventricle infusion, spinal cord, N2a cells
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